The advent of the Joint Clinical Assessment (JCA) process in the European Union, designed to harmonize the clinical evaluation of medicinal products, faces significant procedural and methodological challenges as its first reports loom. While the ambition is to streamline market access and accelerate patient access to innovative therapies, industry stakeholders and experts are raising concerns regarding tight timelines, stringent evidence requirements, and the potential for nationally fragmented implementation. The recent European Commission (EC) approval of Ipsen’s Ojemda (tovorafenib) for paediatric low-grade glioma, marking the first instance to trigger the JCA’s 30-day official endorsement period, underscores the urgency and complexity of this new regulatory landscape.
The JCA Framework: Aims and Early Implementation
Launched in January 2025 under the Health Technology Assessment Regulation (HTAR) [EU 2021/2282], the JCA represents a pivotal shift in how new medicines are assessed across EU member states. The core objective is to create a unified, scientific report on the relative clinical benefit of new technologies, thereby reducing duplicative efforts by national Health Technology Assessment (HTA) bodies. This consolidated assessment is intended to inform national reimbursement and pricing decisions. Initially focused on oncology treatments and advanced therapy medicinal products (ATMPs), the JCA’s scope is slated for expansion to encompass all other product categories in the coming years.
Dr. Alexander Natz, Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), voiced an industry perspective that has generally been positive towards the JCA’s goal of a "one-stop shop" for clinical evaluation. Similarly, Dr. Sreeram Ramagopalan, Senior Lecturer at the Centre for Pharmaceutical Medicine Research at King’s College London, acknowledged the validity of harmonizing clinical assessment, noting that the historical fragmentation of HTA requirements has led to uneven patient access across the EU.
However, the practical implementation of the JCA is revealing divergences from its original aspirations. Patrick Hopkinson, founder and managing director of PHTA Consulting, highlighted that the HTAR, developed over several years through numerous iterations between the EC and Member States, ultimately fell short of industry expectations. A key point of contention is the "voluntary" rather than "mandatory" nature of JCA report uptake by national HTA bodies, a concession that raises questions about the extent of genuine harmonization. The EC has clarified that JCAs will not provide "any value judgements or conclusions on reimbursement," leaving the ultimate responsibility for cost-effectiveness evaluations to individual member states based on their regional needs.
Procedural Bottlenecks: Tight Timelines and Complex PICO Definitions
A significant concern raised by industry experts revolves around the procedural complexities and demanding timelines inherent in the JCA process. The JCA assessment runs in parallel with the European Medicines Agency’s (EMA) review of a drug’s Marketing Authorisation Application (MAA). The process initiates with a JCA scoping phase, where Member States define their national clinical evaluation parameters structured around the PICO framework: Population, Intervention, Comparator, and Outcomes. The appointed assessor and co-assessor are then tasked with consolidating these into a definitive set of PICOs that will govern the assessment.
A critical bottleneck emerges with the requirement for health technology developers to submit a comprehensive dossier within a mere 100 days of scope confirmation by the JCA subgroup. Hopkinson describes this as a "tight timeline, which poses a substantial challenge for a developer." Anecdotal reports, as shared by Dr. Natz, suggest that dossiers can reach up to 30,000 pages, placing a considerable burden on company resources, particularly for smaller and medium-sized enterprises (SMEs).
This compressed timeline is exacerbated by the intricate nature of the evidence required. "An increased risk of avoidable mistakes in the dossier preparation may jeopardize a product’s review outcome despite clinical benefit," warned Dr. Natz. The complexity is further amplified by divergent PICO requests from different Member States, necessitating extensive and intricate analyses. A prime example is the selection of comparator products. While the standard of care can vary significantly between countries, the demand for multiple, potentially politically influenced comparator choices risks overburdening developer resources and diluting the objective of a unified report. Dr. Natz advocates for a delicate balance between the diverse needs of Member States and the practicalities of evidence generation.
Matias Olsen, Senior Manager, Public Affairs & Policy at EUCOPE, noted that while dialogue exists at the national level, the opportunities for direct discussion within the JCA process itself are currently limited. This lack of open communication channels can hinder the ability of companies to adequately prepare and submit the necessary data. Dr. Ramagopalan observed that if divergent PICO requests necessitate the generation of new data, companies may face delays in the JCA process. To mitigate this, Hopkinson stated that companies are often compelled to prepare a substantial portion of the evidence "at risk" even before scope confirmation, with some developers anticipating comparative data requirements related to PICOs a year or two in advance, as added by Ramagopalan.
Stringency of Evidence Requirements: A Conservative Approach
The evidence generation framework for the JCA is heavily influenced by the German HTA approach, characterized by its stringent standards. This includes a cautious stance towards real-world evidence (RWE), single-arm studies, surrogate endpoints, and indirect treatment comparisons. The EC’s JCA guidance explicitly states that evidence generated outside of a randomized controlled trial (RCT) has "much greater potential to include material bias in the estimate of treatment effect."
This conservative approach presents a significant challenge for treatments targeting rare and ultra-rare diseases, where the limited patient population often makes conducting traditional RCTs infeasible or unethical. In such cases, there is a greater reliance on RWE. Paolo Morgese, Vice President of Public Affairs Europe for the Alliance for Regenerative Medicine, emphasized this point, noting the crucial role of RWE for these patient groups. With the JCA’s expansion to include orphan drugs in 2028, followed by all other new medical products in 2030, the current evidence requirements could pose a substantial barrier to access for these specialized therapies.
Both Morgese and Ramagopalan expressed a hope for a more pragmatic assessment approach that deviates from the strictures outlined in the current guidelines. Olsen further stressed the need for clearer direction, arguing that companies are often left "in the dark" when planning their studies. He pointed to the UK’s National Institute for Health and Care Excellence (NICE) as an example of a regulatory body that provides comprehensive guidance on the use and acceptance of real-world data, a model that could benefit the JCA process.
Nationally Fragmented Uptake: The Challenge of Harmonized Reimbursement
The integration of JCA reports into national reimbursement processes across the EU remains a critical area of uncertainty. While the EC’s guidance, published in 2024, mandates that Member States must give "due consideration to the published JCA reports," this leaves considerable discretion to individual countries. Dr. Natz reiterated the industry’s preference for mandatory uptake, stating, "To avoid duplication of efforts, we would have loved to see a mandatory uptake." Despite this, he remains optimistic that Member States will leverage the JCA reports, given their investment of time and resources in the process.
However, Morgese expressed a more reserved outlook regarding the universal adoption of JCA reports into national HTA processes. While countries such as France, Germany, Norway, and the Netherlands have established policies for coordinating the JCA with their national HTA submissions, others, including Romania, Greece, and Hungary, have yet to issue clear payer guidance on how these reports will be incorporated, based on publicly available documents.
The ultimate impact of the JCA on local-level pricing and reimbursement decisions will take time to fully ascertain, according to Hopkinson. The lack of a uniform approach to incorporating JCA findings could undermine the very goal of harmonization, potentially leading to a continuation of fragmented patient access across the EU.
Future Directions: A Call for Urgency and Adaptation
The European Commission is scheduled to conduct a formal review of the JCA process in 2028. However, industry stakeholders argue that significant changes are needed much sooner. Dr. Natz emphasized the urgency, stating, "Companies are deciding whether to launch in Europe in the next few years, and we need to convince those companies now."
Olsen echoed the sentiment for increased urgency, particularly in facilitating sufficient time for companies to interact with assessors. Despite these concerns, he noted that both the EC and the Member State Coordination Group on Health Technology Assessment (HTACG) appear open to receiving feedback, suggesting a willingness for adaptation.
If implemented effectively, the JCA holds the potential to establish a unified voice on the clinical effectiveness of new products within the EU. However, as Morgese cautioned, there is a palpable risk that without careful refinement and adaptation, the JCA could devolve into a bureaucratic exercise, adding an unnecessary layer of complexity to an already intricate regulatory system. The initial experiences with Ojemda and the impending assessments for other innovative medicines will provide crucial early indicators of the JCA’s ability to deliver on its promise of accelerated and equitable patient access across Europe.
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